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Biomarkers for drug development in propionic and methylmalonic acidemias

There is an unmet need for the development and validation of biomarkers and surrogate endpoints for clinical trials in propionic acidemia (PA) and methylmalonic acidemia (MMA). This review examines the pathophysiology and clinical consequences of PA and MMA that could form the basis for potential bi...

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Detalles Bibliográficos
Autores principales:	Longo, Nicola, Sass, Jörn Oliver, Jurecka, Agnieszka, Vockley, Jerry
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	John Wiley & Sons, Inc. 2022
Materias:	Review Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9303879/ https://www.ncbi.nlm.nih.gov/pubmed/35038174 http://dx.doi.org/10.1002/jimd.12478

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